A new therapeutic strategy against prion diseases shows promising results in animal studies.
Autores: Hasier Eraña Un reciente estudio científico, desarrollado por la compañía Sangamo Therapeutics, explora una innovadora terapia génica que ha demostrado ser capaz de retrasar significativamente la progresión de la enfermedad priónica en ratones y de alcanzar todo el cerebro en primates. ¿En qué consiste esta nueva terapia? Las enfermedades priónicas, como la enfermedad de […]
A new breakthrough in detecting diseases such as Parkinson's using a simple skin or nasal mucosa sample
Autores: Tomás Barrio y Alvina Huor Un estudio reciente abre la puerta a diagnósticos más rápidos, accesibles y precisos de enfermedades neurodegenerativas como la enfermedad de Parkinson, la demencia con cuerpos de Lewy y la atrofia multisistémica. Diagnosticar con certeza enfermedades como la enfermedad de Parkinson o la demencia con cuerpos de Lewy sigue siendo […]
We have an IND (Investigational New Drug) open, what's next? A new article on the CureFFI blog by Eric Minikel
Author: Hasier Eraña From the laboratory of Sonia Vallabh and Eric Minikel, at the Broad Institute in Boston, comes news that Eric himself reports on his blog (cureffi.org). On 14 April, these researchers achieved a rare milestone in academic research (that which is done outside of the industry), which is a very rare achievement in [...]
Absence of transmission of chronic wasting disease (CWD) prions to human brain organoids
Author: Enric Vidal One of the many unanswered questions in the field of animal prion diseases is whether or not the prions that cause Chronic wasting disease (CWD) are capable of infecting humans. After the jump from [...]
Towards early detection: Positive RT-QuICR is associated with slower disease progression
Author: Maitena San Juan Creutzfeldt-Jakob neurodegenerative disease (CJD) is a prion disorder caused by the misfolded form of the cellular prion protein (PrPC), also known as PrPSc. Its most common variant is the sporadic form (sCJD), which accounts for approximately 90% of the cases of prion disease diagnosed in humans and is of [...]
Mood disturbances could be an early sign of Creutzfeldt-Jakob disease
Author: Eva Fernández-Muñoz Creutzfeldt-Jakob disease (CJD) is a rare and rapidly progressive neurodegenerative disorder caused by the misfolding of the cellular prion protein (PrPC) into a misfolded form called PrPSc, which accumulates in the brain, causing neuronal damage and spongiform degeneration. The sporadic form (sCJD) is the most common form, accounting for approximately [...]
The Spanish Foundation for Prion Diseases submits its application to the Ministry of Health for the inclusion of prion diseases in the ELA Act.
Author: Joaquín Castilla An essential step in the defence of the rights of patients and their families In an effort to guarantee the recognition and protection of the rights of people affected by prion diseases, the Spanish Foundation for Prion Diseases has exercised its right to participate in the Prior Public Consultation [...]
New strategies to understand and combat sporadic Creutzfeldt-Jakob disease through integration of genetic data
Author: Nuno Anjo Sporadic Creutzfeldt-Jakob disease (sCJD) is a rare, fatal and rapidly progressive neurodegenerative disease for which there are still no effective treatments. It is characterised by the misfolding of the normal prion protein (PrPC) into an abnormal form called PrPSc or Prion, which accumulates in the brain, causing neuronal damage [...].
ION717 clinical trial completes patient enrolment
Author: Eric Minikel (translated by Joaquín Castilla) Ionis Pharmaceuticals has announced (see original letter) this morning that it has completed enrolment of PrProfile, its Phase 1/2a clinical trial of ION717, an ASO drug designed to reduce PrP levels in prion diseases. The study is now listed as "Active, without [...]
Scientists discover early brain alterations in people at risk of developing hereditary Creutzfeldt-Jakob disease
Author: Cristina Sampedro Prion diseases can occur spontaneously or genetically, the latter being caused by a mutation in the prion protein (PrP), which are rare but devastating neurodegenerative disorders that affect both humans and animals. The main types of genetic prion diseases are genetic Creutzfeldt-Jakob disease (gCJD), genetic Creutzfeldt-Jakob disease (gCJD), genetic Creutzfeldt-Jakob disease (gCJD), genetic [...]
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